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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01348087




Registration number
NCT01348087
Ethics application status
Date submitted
3/05/2011
Date registered
5/05/2011
Date last updated
25/05/2016

Titles & IDs
Public title
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
Scientific title
An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
Secondary ID [1] 0 0
2011-001952-12
Secondary ID [2] 0 0
CAFQ056B2279
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fragile X Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AFQ056

Experimental: AFQ056 100 mg (Bid) - All patients initiated treatment with AFQ056 at a starting dose of 25 milligram (mg) twice daily. The dose was titrated from 25mg bid to 50mg bid, 75mg bid and 100mg bid at weekly intervals. Dose adjustments (up- and down titrations) were permitted as needed to manage any tolerability issues and to ensure that patients reach their highest tolerated dose not to exceed 100mg bid.


Treatment: Drugs: AFQ056
The investigational drug, AFQ056, will be provided as hard gelatin capsules. Two different oral dosage strengths, identical in appearance, will be used.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). - Adverse events were summarized for the open-label treatment period, where the open-label treatment period is defined based on how AEs were collected and reported according to the manner in which patients entered the current study and which treatment (AFQ056 or placebo) they were receiving in the previous study. AEs which were continuing from the core study or that started after the end of core study but prior to first dose of open-label study medication in the extension study for Category 1 patients are shown under ('Prior to Ext. first dose'). AEs which started during the open-label treatment period are presented based on the last AFQ056 dose taken on or before the onset date of the AE (25 mg bid; 50 mg bid; 75 mg bid; or 100 mg bid). No efficacy data presented as study was terminated
Timepoint [1] 0 0
Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial

Eligibility
Key inclusion criteria
Inclusion criteria Group 1 patients

- Had to have completed the CAFQ056A2212 study or another study of AFQ056 which included
adult FXS patients within one week of enrollment into the open-label study.

- Females of child-bearing potential had to follow protocol requirements with respect to
contraception.

- Have a caregiver or caregivers who spent, on average, at least six hours per day with
the patient, who were willing and capable of supervising treatment, providing input
into efficacy and safety assessments, and accompanying the patient to study visits.

Group 2:

- Had to have:

- Completed Study CAFQ056A2204.

- Completed Study CAFQ056A2212 or another study of AFQ056 which included adult patients
with FXS but enrollment into the current study was delayed for more than a week.

- Discontinued prematurely from Study CAFQ056A2212 or another study of AFQ056 which
included adult patients with FXS due to intolerability of the dosage in the patient's
assigned treatment group.

- Females of child-bearing potential had to follow protocol requirements with respect to
contraception.

- Have a caregiver or caregivers who spent, on average, at least six hours per day with
the patient, who were willing and capable of supervising treatment, providing input
into efficacy and safety assessments, and accompanying the patient to study visits
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria

Any advanced, severe or unstable disease

- History of severe self- injurious behavior

- History of uncontrolled seizure disorder or resistant to therapy within the past 2
years (Patients who are clinically stable under anti-convulsant therapy for the past 2
years are not excluded)

- History of clinically significant allergies requiring hospitalization or non- inhaled
corticosteroid therapy (asthma, anaphylaxis, etc.)

- Using (or used within 6 weeks before baseline) concomitant medications that are potent
inhibitors or inducers of CYP3A4

- Using glutamatergic agents (riluzole, memantine, etc.) or lithium, digoxin, or
warfarin within 6 weeks of baseline Other protocol-defined inclusion/exclusion
criteria may apply

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Ryde
Recruitment hospital [2] 0 0
Novartis Investigative Site - Waratah
Recruitment hospital [3] 0 0
Novartis Investigative Site - Caulfield
Recruitment postcode(s) [1] 0 0
2112 - Ryde
Recruitment postcode(s) [2] 0 0
2298 - Waratah
Recruitment postcode(s) [3] 0 0
3161 - Caulfield
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Nebraska
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Tennessee
Country [11] 0 0
Canada
State/province [11] 0 0
Ontario
Country [12] 0 0
Canada
State/province [12] 0 0
Quebec
Country [13] 0 0
Denmark
State/province [13] 0 0
Glostrup
Country [14] 0 0
France
State/province [14] 0 0
Bron Cedex
Country [15] 0 0
France
State/province [15] 0 0
Paris
Country [16] 0 0
Germany
State/province [16] 0 0
Berlin
Country [17] 0 0
Germany
State/province [17] 0 0
Mainz
Country [18] 0 0
Germany
State/province [18] 0 0
Tübingen
Country [19] 0 0
Germany
State/province [19] 0 0
Würzburg
Country [20] 0 0
Italy
State/province [20] 0 0
GE
Country [21] 0 0
Spain
State/province [21] 0 0
Andalucia
Country [22] 0 0
Spain
State/province [22] 0 0
Catalunya
Country [23] 0 0
Switzerland
State/province [23] 0 0
Lausanne
Country [24] 0 0
Switzerland
State/province [24] 0 0
Zurich
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Edinburgh

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to generate long-term safety, tolerability and efficacy data for
AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212
(NCT01253629).study and patients who have participated in the previous proof-of-concept study
CAFQ056A2204 (NCT00718341).
Trial website
https://clinicaltrials.gov/show/NCT01348087
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT01348087